Press Release

CRISPR Therapeutics obtains an exclusive license to use Lipid Nanoparticle technologies for in vivo CRISPR/Cas9-based therapies

BASEL, Switzerland and CAMBRIDGE, Mass., May 08, 2017 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ:CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases today announced they have signed an exclusive license with the Massachusetts Institute of Technology (MIT) for a family of Lipid Nanoparticle (LNP) technologies developed in the lab of Dr. Daniel G. Anderson, a scientific founder and advisory board member of CRISPR Therapeutics.

Under the terms of the license, CRISPR Therapeutics obtains the exclusive rights to use the LNP technologies in their therapeutic development programs focused on in vivo gene editing applications. MIT receives an upfront technology access fee, milestones, and royalties on licensed products that reach the market.

“We have been working to evaluate a panel of different LNP technologies for in vivo editing applications based on their potency and tolerability. These specific compounds from Dr. Anderson’s lab continue to perform well relative to others we have tested, and we are excited to sign this license with MIT. The primary focus here is liver indications, where we are continuing to optimize our platform for both gene disruption and correction, and have demonstrated very potent gene disruption in murine liver,” said Dr. Chad Cowan, Head of Research at CRISPR Therapeutics.

Dr. Anderson is an Associate Professor of Chemical Engineering and the Institute for Medical Engineering and Science, and member of the Koch Institute for Integrative Cancer Research at MIT. He is a world-leader in the development of advanced drug delivery systems for non-viral gene delivery. He was also one of the first to demonstrate the potential for in vivo gene editing using CRISPR/Cas9.

“Our laboratory has worked to develop non-viral delivery vectors for nucleic acids, and we are finding that LNP technologies work very well for delivering the CRISPR/Cas9 system. In fact, gene editing applications may be particularly well-suited to LNP delivery given the potential for single dose efficacy. I am excited to see CRISPR-based in vivo therapies moving rapidly toward the clinic,” said Dr. Daniel Anderson.

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene-editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR / Cas9 gene-editing platform. CRISPR / Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. The company's multi-disciplinary team of world-class researchers and drug developers is working to translate this technology into breakthrough human therapeutics in a number of serious diseases. Additionally, CRISPR Therapeutics has established strategic collaborations with Bayer AG and Vertex Pharmaceuticals to develop CRISPR-based therapeutics in diseases with high unmet need. The foundational CRISPR / Cas9 patent estate for human therapeutic use was licensed from the company's scientific founder Emmanuelle Charpentier, Ph.D. CRISPR Therapeutics AG is headquartered in Basel, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts. For more information, please visit http://www.crisprtx.com.

CRISPR Forward-Looking StatementCertain statements set forth in this press release constitute “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, but not limited to, statements concerning: the therapeutic value, development, and commercial potential of CRISPR/Cas-9 gene editing technologies and therapies and the intellectual property protection of our technology and therapies. You are cautioned that forward-looking statements are inherently uncertain. Although the company believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, the forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: uncertainties inherent in the initiation and completion of preclinical and clinical studies for the Company’s product candidates; uncertainties regarding the intellectual property protection for our technology and intellectual property belonging to third parties; availability and timing of results from preclinical and clinical studies; whether results from a preclinical trial will be predictive of future results of the future trials; expectations for regulatory approvals to conduct trials or to market products; and those risks and uncertainties described in Item 1A under the heading “Risk Factors” in the company’s most recent annual report on Form 10-K, and in any other subsequent filings made by the company with the U.S. Securities and Exchange Commission (SEC), which are available on the SEC’s website at www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made.